– Primary and secondary endpoints were met, key
exploratory endpoints supported clinical benefit in Phase 3 study
of omidubicel in patients with hematologic malignancies; BLA
submission anticipated in fourth quarter of 2021-
— Omidubicel commercial preparation underway,
including the creation of Gamida Cell Assist, to support potential
launch —
– GDA-201 demonstrated significant clinical
activity in Phase 1 study of patients with non-Hodgkin lymphoma,
with multiple complete responses observed; Phase 1/2 clinical trial
planned with IND submission anticipated in the second half of
2021—
– Strengthened financial position with sale of
$75M ordinary shares in December 2020 and $75M exchangeable senior
notes in February 2021; sufficient liquidity to fund the company’s
operations into the second half of 2022 —
– Company to host conference call at 8:30 a.m.
ET today –
Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy
company committed to finding cures for blood cancers and serious
blood diseases, today reported financial results for the year and
quarter ended December 31, 2020. The company also highlighted
progress with omidubicel, an advanced cell therapy in Phase 3
clinical development as a potentially life-saving treatment option
for patients in need of bone marrow transplant, and GDA-201, a
natural killer (NK) cell immunotherapy in Phase 1 development in
patients with non-Hodgkin lymphoma (NHL).
“It has been a significant year for Gamida Cell, marked by a
number of important achievements that have brought us closer to
developing cures for blood cancers and serious hematologic
diseases. Omidubicel, an advanced cell therapy that has met all
primary, secondary and exploratory endpoints in our Phase 3 study
in patients with hematological malignancies, represents a
potentially transformative treatment option. The data we presented
in 2020, demonstrating the clinical benefit of omidubicel, position
us to submit our first BLA for omidubicel in the fourth quarter of
2021,” said Julian Adams, Ph.D., chief executive officer of Gamida
Cell. “We remain focused on both the BLA submission and preparing
for potential commercial readiness, with the announcement of Gamida
Cell Assist, a program designed to focus on patient access and a
positive omidubicel experience for the patient and the transplant
team. We are diligently working to bring omidubicel to patients as
soon as possible.”
“We believe our NAM-based cell expansion technology has
potential for NK cell expansion and we are developing GDA-201, an
NK-cell immunotherapy for the treatment of hematologic and solid
tumors in combination with antibody therapies. This year, we made
meaningful progress with GDA-201, which has demonstrated impressive
early results in patients with heavily pre-treated NHL in a Phase 1
investigator-sponsored study. Following these results, we plan to
submit an IND to the FDA in the second half of 2021 and initiate a
Phase 1/2 study,” Dr. Adams continued.
Omidubicel, an investigational advanced cell therapy for
allogeneic bone marrow transplant
During the year, Gamida Cell made significant progress to
advance its Phase 3 product candidate omidubicel, which is the
first cell therapy for bone marrow transplant to receive
Breakthrough Therapy Designation from the U.S. Food and Drug
Administration (FDA) and which has the potential to be the first
FDA-approved engineered cell therapy which can be used as a bone
marrow transplant graft. The company presented primary, secondary
and exploratory endpoints from the company’s international,
multi-center, randomized Phase 3 study of omidubicel demonstrating
its clinical benefit as a treatment option for patients in need of
a bone marrow transplant.
In May, Gamida Cell reported that the phase 3 study of
omidubicel achieved its primary endpoint, demonstrating a
statistically significant reduction in time to neutrophil
engraftment, a key milestone in recovery from a bone marrow
transplant. It was shown that the median time to neutrophil
engraftment was 12 days for patients randomized to omidubicel
compared to 22 days for the comparator group (p<0.001). The
Phase 3 study was designed to evaluate the safety and efficacy of
omidubicel in patients with hematologic malignancies undergoing a
bone marrow transplant compared to a comparator group of patients
who received a standard umbilical cord blood transplant.
The Phase 3 study additionally met key secondary endpoints
related to platelet engraftment, infections and hospitalization,
all significant clinical measures in bone marrow transplant, as
reported in October 2020. The prespecified secondary endpoints,
analyzed in all randomized patients (intent-to-treat), were the
proportion of patients who achieved platelet engraftment by day 42,
the proportion of patients with Grade 2 or Grade 3 bacterial or
invasive fungal infections in the first 100 days following
transplant, and the number of days alive and out of the hospital in
the first 100 days following transplant. All three secondary
endpoints demonstrated a statistically significant improvement
among patients who were randomized to omidubicel compared to the
comparator group.
Recently, the results of the company’s Phase 3 study of
omidubicel were presented at the Transplantation & Cellular
Therapy Meetings of the American Society of Transplantation and
Cellular Therapy and Center for International Blood & Marrow
Transplant Research. The data from the study relating to
exploratory endpoints also supported the clinical benefit
demonstrated by the study’s primary and secondary endpoints. Safety
results were also presented, showing no significant difference
between the two patient groups related to grade III/IV acute GvHD
(14 percent for omidubicel, 21 percent for the comparator) or all
grades chronic GvHD at one year (35 percent for omidubicel, 29
percent for the comparator). Transplants with umbilical cord blood,
the comparator, have been historically shown to result in low
incidence of GvHD in relation to other graft sources, and in this
study, omidubicel demonstrated a similar GvHD profile. The rate of
infection was significantly reduced for patients randomized to
omidubicel, with the cumulative incidence of first grade 2 or grade
3 bacterial or invasive fungal infection for patients randomized to
omidubicel of 37 percent, compared to 57 percent for the comparator
(p = 0.027). Additionally, the study demonstrated a reduction in
the incidence of viral infections. Non-relapse mortality was 11
percent for patients randomized to omidubicel and 24 percent for
patients randomized to the comparator (p=0.09). Overall survival at
one year following transplant was 73 percent for patients
randomized to omidubicel and 62 percent for patients randomized to
control (p=0.16).When considering the patient experience following
transplant, faster hematopoietic recovery, fewer bacterial and
viral infections and fewer days in hospital are all meaningful
results and represent potentially important advancements in
care.
Additional omidubicel highlights:
- Presented new Phase 1 data from study of omidubicel in
patients with severe aplastic anemia (SAA) at ASH: In a poster
presentation at ASH, Gamida Cell presented data demonstrating that
patients with severe aplastic anemia treated with omidubicel
achieved sustained early engraftment and robust immune
reconstitution following reduced intensity conditioning. The data
suggest that omidubicel can result in rapid engraftment and can
achieve sustained hematopoiesis in patients who are at high risk
for graft failure with conventional umbilical cord blood
transplant. The study remains open for accrual of patients with
SAA.
- Advanced commercial launch readiness: Gamida Cell
recently announced plans for the Gamida Cell Assist program. The
transplant process can be challenging and complex for the patient,
caregivers and the entire transplant care team. Gamida Cell Assist
has been designed to focus on patient access and support of every
individual and their caregivers at each step of the process. Once
the program is launched, the Gamida Cell Assist case management
team will provide a consistent, single point of contact for
patients and health care professionals, and work with the
transplant center to track production of omidubicel for each
individual patient and provide real-time updates on the status of
the therapy. The services provided will include coverage and
reimbursement support, which may include financial, travel, and
lodging assistance. Gamida Cell is committed to supporting a
positive journey for patients and their transplant teams so they
can focus on what matters most – the patient experience and
successful clinical outcomes.
- Expanded collaboration with Be The Match BioTherapies®:
In October, Gamida Cell and Be The Match Therapies® expanded their
existing strategic collaboration for omidubicel. In building upon
the existing collaboration, Gamida Cell will work through Be The
Match BioTherapies® for the supply of cord blood units, which serve
as the starting material for omidubicel. The expanded agreement is
designed to provide a smooth process throughout the omidubicel
therapy supply chain.
GDA-201, an innate NK cell immunotherapy
- Presented updated Phase 1 data at the 62nd ASH Annual
Meeting: In December, Gamida Cell announced updated data from
the ongoing Phase 1 study of GDA-201 in combination with monoclonal
antibodies in patients with NHL and multiple myeloma at the ASH
Annual Meeting. GDA-201 in combination with rituximab demonstrated
significant clinical activity in relapsed and refractory NHL
patients, with 13 complete responses and one partial response
observed in the first 19 NHL patients, for an overall response rate
of 74 percent. Overall survival and progression-free survival at
one year in the NHL cohort suggest durable disease control, with a
median follow-up of ten months (range 1–28 months), in heavily
pretreated patients. Additionally, there were no dose-limiting
toxicities, neurotoxic events, confirmed cytokine release syndrome,
GvHD or marrow aplasia.
- Continued advancing Phase 1 study of GDA-201: Gamida
Cell continues to advance activities to enable the submission of an
investigational new drug (IND) application for cryopreserved,
off-the-shelf GDA-201 to enable a multi-center, Phase 1/2 clinical
study in patients with NHL in the second half of 2021. Gamida Cell
is pioneering a novel approach that harnesses the power of its cell
expansion technology, which uniquely improves antibody-dependent
cellular cytotoxicity and tumor targeting of NK cells.
Corporate Highlights
- Strengthened financial position: In December 2020, the
company executed an underwritten public offering raising
approximately $75 million before deducting underwriting discounts,
commissions and offering expenses. Also, in February 2021, the
company announced a $75 million financing with Highbridge Capital
Management, LLC before deducting offering expenses. These capital
infusions will be used to support manufacturing, regulatory and
potential commercial development activities for omidubicel and to
further the preclinical and clinical development of GDA-201.
Full Year 2020 Financial Results
- Research and development (R&D) expenses in 2020 were $41.4
million, compared to $31.5 million in 2019. The increase was mainly
due to advancing the GDA-201 clinical program and clinical
activities relating to concluding our Phase 3 clinical trial, as
well as additional headcount within the R&D organization.
- Commercial expenses in 2020 were $8.7 million, compared to $4.7
million in 2019. The increase was attributed to an increase in
omidubicel commercial readiness activities as well as additional
headcount within the Commercial organization.
- General and administrative expenses were $12.2 million in 2020,
compared to $12.1 million in 2019. The increase was mainly due to a
$1.3 million increase in professional services expenses, including
Legal and Insurance, offset by decrease of $1.2 million in Travel
and non-cash compensation expenses.
- Finance expenses, net, was $10.4 million for 2020, compared to
finance income, net, of $13.8 million for 2019. The decrease was
primarily due to non-cash expenses resulting from revaluation of
warrants owned by certain of the company’s shareholders and the
revaluation of the Israeli Innovation Authority royalty-bearing
grant liability.
- Net loss for 2020 was $72.7 million, compared to a net loss of
$34.4 million in 2019.
- As of December 31, 2020, Gamida Cell had total cash and cash
equivalents of $127.2 million, compared to $55.4 million as of
December 31, 2019. In addition, on February 16, 2020, Gamida Cell
announced the sale of $75 million exchangeable senior notes due in
2026 to Highbridge Capital Management, LLC.
2020 Financial Guidance
Gamida Cell expects cash used for ongoing operating activities
in 2021 to range from $100 million to $120 million.
Gamida Cell expects that its current cash and cash equivalents
will support the company’s ongoing operating activities into the
second half of 2022. This cash runway guidance is based on the
company’s current operational plans and excludes any additional
funding and any business development activities that may be
undertaken.
Expected 2021 Milestones
Gamida Cell plans to achieve the following milestones during
2021:
Omidubicel
- BLA submission to the FDA in the fourth quarter of 2021
- Commercial readiness activities underway for potential launch
at approval
GDA-201
- Submit company-sponsored IND application to the FDA and
initiate a Phase 1/2 clinical study in NHL in the second half of
2021
Conference Call Information
Gamida Cell will host a conference call today, March 9, 2021, at
8:30 a.m. ET to discuss these financial results and company
updates. A live webcast of the conference call can be accessed in
the “Investors & Media” section of Gamida Cell’s website at
www.gamida-cell.com. To participate in the live call, please dial
866-930-5560 (domestic) or 409-216-0605 (international) and refer
to conference ID number 1996281. A recording of the webcast will be
available approximately two hours after the event, for
approximately 30 days.
About Omidubicel
Omidubicel is an advanced cell therapy under development as a
potential life-saving allogeneic hematopoietic stem cell (bone
marrow) transplant solution for patients with hematologic
malignancies (blood cancers). In both Phase 1/2 and Phase 3
clinical studies (NCT01816230, NCT02730299), omidubicel
demonstrated rapid and durable time to engraftment and was
generally well tolerated.1,2 Omidubicel is also being evaluated in
a Phase 1/2 clinical study in patients with severe aplastic anemia
(NCT03173937). The aplastic anemia investigational new drug
application is currently filed with the FDA under the brand name
CordIn®, which is the same investigational development candidate as
omidubicel. For more information on clinical trials of omidubicel,
please visit www.clinicaltrials.gov.
Omidubicel is an investigational therapy, and its safety and
efficacy have not been established by the FDA or any other health
authority.
About GDA-201
Gamida Cell applied the capabilities of its NAM-based cell
expansion technology to develop GDA-201, an innate NK cell
immunotherapy for the treatment of hematologic and solid tumors in
combination with standard of care antibody therapies. GDA-201
addresses key limitations of NK cells by increasing the
cytotoxicity and in vivo retention and proliferation in the bone
marrow and lymphoid organs of NK cells expanded in culture. GDA-201
is in Phase 1 development through an investigator-sponsored study
in patients with refractory non-Hodgkin lymphoma and multiple
myeloma.3 For more information on the clinical study of GDA-201,
please visit www.clinicaltrials.gov.
GDA-201 is an investigational therapy, and its safety and
efficacy have not been established by the FDA or any other health
authority.
About Gamida Cell
Gamida Cell is an advanced cell therapy company committed to
cures for patients with blood cancers and serious blood diseases.
We harness our cell expansion platform to create therapies with the
potential to redefine standards of care in areas of serious medical
need. For additional information, please visit
www.gamida-cell.com or follow Gamida Cell on LinkedIn
or Twitter at @GamidaCellTx.
Cautionary Note Regarding Forward Looking Statements
This press release contains forward-looking statements as that
term is defined in the Private Securities Litigation Reform Act of
1995, including with respect to timing of initiation and progress
of and data reported from the clinical trials of Gamida Cell’s
product candidates, anticipated regulatory filings,
commercialization efforts and Gamida Cell’s expectations regarding
its projected ongoing operating activities and cash runway, which
statements are subject to a number of risks, uncertainties and
assumptions, including, but not limited to the scope, progress and
expansion of Gamida Cell’s clinical trials and ramifications for
the cost thereof; and clinical, scientific, regulatory and
technical developments. In light of these risks and uncertainties,
and other risks and uncertainties that are described in the Risk
Factors section and other sections of Gamida Cell’s Annual Report
on Form 20-F, filed with the Securities and Exchange Commission
(SEC) on March 9, 2021 and other filings that Gamida Cell makes
with the SEC from time to time (which are available at
http://www.sec.gov), the events and circumstances discussed in such
forward-looking statements may not occur, and Gamida Cell’s actual
results could differ materially and adversely from those
anticipated or implied thereby. Any forward-looking statements
speak only as of the date of this press release and are based on
information available to Gamida Cell as of the date of this
release.
CONSOLIDATED STATEMENTS OF FINANCIAL
POSITION U.S. dollars in thousands
December 31,
2020
2019
ASSETS
CURRENT ASSETS:
Cash and cash equivalents
$
127,170
$
41,838
Marketable securities
-
13,559
Prepaid expenses and other current
assets
2,815
1,306
Total current assets
129,985
56,703
NON-CURRENT ASSETS:
Property, plant and equipment, net
18,238
6,298
Right-of-use assets
6,474
5,133
Other assets
786
641
Total non-current assets
25,498
12,072
Total assets
$
155,483
$
68,775
LIABILITIES AND SHAREHOLDERS' EQUITY
CURRENT LIABILITIES:
Trade payables
$
6,329
$
1,164
Employees and payroll accruals
4,705
3,443
Current maturities of lease
liabilities
2,532
1,870
Accrued expenses and other payables
7,988
4,918
21,554
11,395
NON-CURRENT LIABILITIES:
Liabilities presented at fair value
12,043
5,221
Employee benefit liabilities, net
768
773
Lease liability
5,378
4,101
Liability to Israel Innovation Authority
(IIA)
17,003
12,302
35,192
22,397
CONTINGENT LIABILITIES AND COMMITMENTS
SHAREHOLDERS' EQUITY:
Share capital
166
92
Share premium
375,280
238,992
Capital reserve due to actuarial loss
(441
)
(541
)
Reserve from financial assets measured at
FVOCI
-
4
Accumulated deficit
(276,268
)
(203,564
)
Total shareholders' equity
98,737
34,983
Total liabilities and shareholders'
equity
$
155,483
$
68,775
CONSOLIDATED STATEMENTS OF
COMPREHENSIVE LOSS U.S. dollars in thousands (except share and
per share data)
Year ended December
31,
2020
2019
2018
Operating expenses:
Research and development expenses, net
$
41,385
$
31,462
$
22,045
Commercial activities
8,748
4,692
-
General and administrative expenses
12,167
12,091
11,599
Operating loss
62,300
48,245
33,644
Financial expenses
10,640
3,325
20,259
Financial income
(236
)
(17,149
)
(1,042
)
Loss before taxes on income
72,704
34,421
52,861
Taxes on income
-
(70
)
70
Net loss
72,704
34,351
52,931
Net loss per
share:
Basic net loss per share
$
1.66
$
1.17
$
10.53
Diluted net loss per share
$
1.66
$
1.69
$
10.53
CONSOLIDATED STATEMENTS OF CASH
FLOWS U.S. dollars in thousands
Year ended December
31,
2020
2019
2018
Cash flows from
operating activities:
Net loss
$
(72,704
)
$
(34,351
)
$
(52,931
)
Adjustments to reconcile net loss to net
cash used in operating activities:
Adjustments to the profit or loss
items:
Depreciation of property, plant and
equipment and right-of-use assets
2,397
2,143
269
Financial loss (income), net
483
(775
)
(858
)
Share-based compensation
2,864
4,868
3,575
Change in employee benefit liabilities,
net
94
126
(15
)
Amortization of premium on marketable
securities
4
184
272
Revaluation of financial derivatives
6,822
(15,904
)
17,600
Revaluation of liability to IIA
4,302
2,531
2,037
16,966
(6,827
)
22,880
Changes in asset and liability items:
Increase (decrease) in other receivables,
prepaid expenses and other assets
(1,626
)
(150
)
942
Increase (decrease) in trade payables
5,083
(821
)
(405
)
Increase in accrued expenses and other
payables
3,454
2,807
2,296
6,911
1,836
2,833
Cash received during the year for:
Interest received
361
1,546
792
Interest paid
(161
)
(134
)
-
Net cash used in operating activities
(48,627
)
(37,930
)
(26,426
)
Cash flows from
investing activities:
Purchase of property, plant and
equipment
(11,804
)
(3,055
)
(1,645
)
Purchase of marketable securities
-
(32,021
)
(10,905
)
Proceeds from bank deposits
-
-
5,000
Investment in restricted bank deposits
(158
)
-
(150
)
Proceeds from maturity of marketable
securities
13,551
38,742
-
Proceeds from sale of marketable
securities
-
-
4,949
Net cash provided by (used in) investing
activities
1,589
3,666
(2,751
)
CONSOLIDATED STATEMENTS OF CASH
FLOWS U.S. dollars in thousands
Year ended December
31,
2020
2019
2018
Cash flows from
financing activities:
Proceeds from secondary offerings, net
133,316
37,140
-
Receipt of grants from the IIA
399
224
612
Proceeds from issuance of shares, initial
public offering (payment of issuance expenses), net
-
(238)
47,479
Payment of lease liabilities
(1,985)
(1,529)
-
Proceeds from exercise of options
650
132
2
Net cash provided by financing
activities
132,380
35,729
48,093
Exchange differences on balances of cash
and cash equivalents
(10)
101
31
Increase in cash and cash equivalents
85,332
1,566
18,947
Cash and cash equivalents at beginning of
year
41,838
40,272
21,325
Cash and cash equivalents at end of
year
$ 127,170
$ 41,838
$ 40,272
1 Horwitz M.E., Wease S., Blackwell B., Valcarcel D. et al.
Phase I/II study of stem-cell transplantation using a single cord
blood unit expanded ex vivo with nicotinamide. J Clin Oncol. 2019
Feb 10;37(5):367-374. 2 Gamida Cell press release, “Gamida Cell
Announces Positive Topline Data from Phase 3 Clinical Study of
Omidubicel in Patients with High-Risk Hematologic Malignancies,”
issued May 12, 2020. Last accessed August 31, 2020. 3
Clinicaltrials.gov identifier NCT03019666
View source
version on businesswire.com: https://www.businesswire.com/news/home/20210309005258/en/
For investors: Stephanie Ascher Stern Investor Relations, Inc.
stephanie.ascher@sternir.com 1-212-362-1200 For media: Rhiannon
Jeselonis Ten Bridge Communications
rhiannon@tenbridgecommunications.com 1-978-417-1946
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